Open Access

Patient access schemes in Asia-pacific markets: current experience and future potential

  • Christine Y Lu1Email author,
  • Caitlin Lupton1,
  • Shana Rakowsky2,
  • Zaheer-Ud-Din Babar3,
  • Dennis Ross-Degnan1 and
  • Anita K Wagner1
Journal of Pharmaceutical Policy and Practice20158:6

https://doi.org/10.1186/s40545-014-0019-x

Received: 8 September 2014

Accepted: 4 December 2014

Published: 16 February 2015

Abstract

Objectives

Patient access (or risk-sharing) schemes are alternative market access agreements between healthcare payers and medical product manufacturers for conditional coverage of promising health technologies. This study aims to identify and characterize patient access schemes to date in the Asia-Pacific region.

Methods

We reviewed the literature on patient access schemes over the last two decades using publicly available databases, Internet, and grey literature searches. We extracted key features of each scheme identified, including the drug, clinical indication, stakeholders involved, and details of the scheme. We categorized schemes according to a previously published taxonomy of scheme types and by country.

Results

We identified 3 schemes in South Korea, 5 in New Zealand, and 98 in Australia. Most (97.2%; n = 103) schemes focused on pharmaceuticals, few on medical technologies. More than half of the schemes related to treatments for cancer and inflammatory diseases such as rheumatoid arthritis. The majority (77.4%; n =82) involved pricing arrangements. Evidence generation schemes were rarely used. About half (41.8%; n = 41) of schemes in Australia were hybrid by nature, consisting of pricing arrangements with a conditional treatment continuation component.

Conclusions

Australia has the most experience with patient access schemes and its experience may provide useful insights for other Asia-Pacific countries. The main targets are pharmaceuticals likely to have high budget impact (due to high per-patient costs and/or large volumes of use), and pharmaceuticals that may be adopted more widely than indicated. With the proliferation of high-cost medicines, the use of schemes may increase to address rising cost pressures, consumer demands, and uncertainties, while attempting to provide patient access to innovative care within finite budgets. Future research is warranted to evaluate the performance of patient access schemes.

Keywords

Patient access schemes Risk sharing Managed entry Conditional coverage Health technology assessment High cost medicines Access to medicines

Introduction

Many countries and insurance schemes are pursuing universal health coverage with the goal of ensuring that all people have access to needed health services without suffering financial hardship [1,2]. However, healthcare systems grapple with the challenges of funding clinically beneficial health services and medicines while ensuring that financing systems are sustainable.

In an effort to improve the efficiency of rising healthcare expenditures, many systems evaluate whether expected additional health benefits of a new technology justify its additional cost compared to existing treatments (cost-effectiveness or ‘value-for-money’) [3-5]. Healthcare systems in the Asia-Pacific region that require proof of value-for-money in coverage of medical technologies include Australia, New Zealand, Thailand, South Korea, and Taiwan [6-10]. Even in systems that do not explicitly consider cost, there is often a focus on the magnitude of health benefits, which are informally weighed against cost. Traditionally, coverage decisions on medical technologies such as medicines and devices have been binary in nature; based on evidence available at launch and the price set by the manufacturer, payers decide whether or not to reimburse a product. In recent years, various types of conditional coverage decisions have emerged. Patient access schemes (also known as managed entry schemes or risk-sharing agreements) are alternative market access agreements, typically between payers and manufacturers, to enable provisional or conditional coverage of promising health technologies [4,5,11]. There are three broad categories of schemes [5]. First, outcome-based schemes (also known as performance-based or effectiveness guarantee schemes) in which the price, level, or nature of reimbursement are tied to clinical or intermediate endpoints measured in the future and ultimately related to patients’ quality or quantity of life. Second, evidence generation schemes in which a positive coverage decision is conditioned upon the collection of additional evidence through clinical studies, which might result in continued, expanded, or withdrawn coverage. Third, financially-based schemes negotiate company contributions to the cost of a product (e.g., discounts or rebates, price-volume agreements, utilization caps) for a particular patient or population without linking reimbursement to health outcomes.

Interest in patient access schemes is growing due to increasing cost pressures, the need to balance the interests of patients, clinicians, manufacturers, and other stakeholders, and uncertainties due to incomplete information at the time that decisions must be made. The necessary evidence to demonstrate incremental effectiveness and cost-effectiveness of a new technology is often not available at the time of launch [12]. Therefore, uncertainties exist about whether the technology will deliver the promised health gains in routine clinical practice. Clinical and cost-effectiveness uncertainties result in budget uncertainties [12]. In addition, costs of innovative products may bankrupt households and threaten sustainability of systems. Patient access schemes offer an important option for systems to allow (some) patients (early) access to promising technologies that systems may not otherwise fund. At the same time, these schemes may reduce the financial risks that payers face in making decisions based on limited evidence, and in some cases, they facilitate collection of more evidence to support future decisions [4,5,11].

Little is known about experiences with patient access schemes in Asia-Pacific countries. The purpose of this study was to identify and categorize patient access schemes in this region. The results of this study will enhance understanding of the roles of patient access schemes in emerging Asia-Pacific markets and inform policy developments for enabling patient access to promising technologies.

Methods

We reviewed the literature on patient access schemes. We searched in PubMed and Google Scholar for English-language articles published through July 2012 using the following terms; ‘conditional coverage’ , ‘conditional reimbursement’ , ‘managed entry’ , ‘risk sharing’ , ‘risk sharing agreement’ , ‘risk sharing, pharmaceuticals’ , ‘risk sharing scheme’ , ‘coverage with evidence’ , ‘value-based pricing, pharmaceuticals’ , ‘patient access scheme’ , ‘pay back schemes’ , ‘performance-based, pharmaceuticals’ , ‘outcome-based reimbursement, pharmaceuticals’ , ‘reimbursement mechanisms, pharmaceuticals’ , and ‘outcome guarantee’. We scanned titles and abstracts to select relevant articles to review. The reference lists of relevant articles were also reviewed for studies fitting our criteria that our search strategy may have missed. We also added relevant unpublished or ‘grey literature’ (reports, conference presentations, payer websites) that were identified during our search.

We extracted key features of the schemes: the technology, disease area, country, payer, manufacturer, scheme type, and agreement details (when reported). We categorized the schemes according to a published taxonomy [5] of scheme types: (1) outcome-based schemes, (2) evidence generation schemes, and (3) financially-based schemes. These scheme types have generally been termed “risk sharing schemes” or “patient access schemes” by payers or pharmaceutical companies. Consistent with the literature [13], we classified schemes with a “conditional treatment continuation” component that limits continued subsidy of medicines to patients who demonstrate a pre-specified adequate clinical response as outcome-based schemes. We deliberately included financially-based schemes, in contrast to previous reviews of patient access schemes that focused on outcome-based schemes only [13]. Although financially-based schemes do not directly address uncertainties in clinical outcomes, they can address uncertainties in cost-effectiveness and/or budget impact estimates. Their use is increasing rapidly because they may be more feasible than outcome-based schemes that require more information and are administratively burdensome. We did not limit our search to any country. We report on schemes identified in Asia-Pacific markets.

Results

Search results

Using the search terms in PubMed and Google Scholar, we found 2229 articles published between 1998 and July 2012. Reviewing article titles and abstracts, we excluded commentaries and articles that did not describe specific patient access schemes. Next, we reviewed the references of remaining articles and grey literature sources for additional examples of patient access schemes. Our search identified 299 schemes described in 146 publicly available sources (92 scientific articles, 18 electronic articles, 14 reports, 9 websites, and 13 conference presentations). From these we selected the 106 examples from the Asia-Pacific region.

Participating countries

We identified 106 patient access schemes in 3 countries from the Asia-Pacific region (Table 1). Public payers were involved in all schemes. Most (92.5%; n = 98) have taken place in Australia (Table 2) [14-21]. The Australian Pharmaceutical Benefits Advisory Committee (PBAC) is an expert committee that reviews incremental effectiveness and cost-effectiveness of medicines for coverage under the Pharmaceutical Benefits Scheme. The PBAC has formally assessed evidence of cost-effectiveness since 1993 [4]. The Medical Benefits Advisory Committee is an equivalent committee that evaluates medical devices for coverage under the Medical Benefits Scheme.
Table 1

Patient access schemes in the Asia-Pacific region by country, type and condition

 

Australia

South Korea

New Zealand

Total

Types

    

  Outcome-based

21

-

-

21

  Evidence generation

3

-

-

3

  Financially-based

33

3

5

41

  Hybrid*

41

-

-

41

Conditions

  Cancer

29

-

2

31

  Inflammatory Conditions

28

-

1

29

  Infectious Disease

7

-

-

7

  Pulmonary Hypertension

7

1

1

9

  Other

27

2

1

30

Technology

  Pharmaceuticals

95

3

5

103

  Medical devices

3

-

-

3

Subtotal

98

3

5

106

*Hybrid schemes involved both pricing arrangements and conditional treatment continuation

Table 2

Identified patient access schemes in Australia

Technology

Indication

Company

Payer

Type

Source

Cancer (n = 29)

Dasatinib

CML

Bristol-Myers Squibb Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Nilotinib

CML

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Imatinib

GIST, adjuvant

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Imatinib

GIST, metastatic

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Lapatinib

Late stage metastatic breast cancer

GlaxoSmithKline Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Imatinib

Rare cancers (eg dermatofibrosarcoma protuberans, chronic eosinophilic leukemia)

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Pazopanib

RCC

GlaxoSmithKline Australia Pty Ltd

PBS

Financially-based; Outcome-based

Public Summary Document [18]

Bevacizumab

Metastatic colorectal cancerr

Roche Products Pty Ltd

PBS

Financially-based; Outcome-based

Public Summary Document [18]

Sorafenib

Advanced hepatocellular carcinoma

Bayer Australia Ltd

PBS

Financially-based; Outcome-based

Public Summary Document [18]

PET

Staging of newly diagnosed NSCLC, esophageal cancer, cancer of gastro-esophageal junction, head and neck cancer, suspected residual/metastatic/recurrent colorectal cancer, melanoma, head and neck cancer, ovarian cancer

Not specified

MBS

Evidence generation

Stafinski et al. 2010 [15]

Exemestane

Oestrogen-receptor positive advanced breast cancer in post-menopausal women

Pfizer Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Imiquimod

Superficial basal cell carcinoma

3 M Pharmaceuticals Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Letrozole

Advanced breast cancer in postmenopausal women

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Docetaxel

SCCHN

Sanofi-Aventis Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Sunitinib

RCC

Pfizer Australia Pty Ltd

PBS

Financially-based

PBS Schedule [16], Public Summary Document [18]

Cetuximab

K-RAS wild mCRC

Merck Serono Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Vemurafenib

Untreated unresectable stage IIIC or stage IV melanoma

Roche Products Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Imatinib

ALL

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBPA Relativity Sheets [17], Public Summary Document [18], Medicare Australia [14]

Dasatinib

ALL

Bristol-Myers Squibb Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBPA Relativity Sheets [17], Public Summary Document [18], Medicare Australia [14]

Azacitidine

CML

Celgene Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Azacitidine

AML

Celgene Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Azacitidine

Myelodysplastic syndrome

Celgene Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Imatinib

CML

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBPA Relativity Sheets [17], Medicare Australia [14]

Trastuzumab

HER2+ early breast cancer

Roche Products Pty Ltd

PBS

Financially-based; Outcome-based

PBPA Relativity Sheets [17], Medicare Australia [14]

Sunitinib

GIST

Pfizer Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Lenalidomide

Myelodysplastic syndrome

Celgene Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Lenalidomide

Multiple myeloma

Celgene Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Bortezomib

Multiple myeloma

Janssen-Cilag Pty Ltd

PBS

Financially-based, Outcome-based

PBS Schedule [16], PBPA Relativity Sheets [17], Public Summary Document [18], Medicare Australia [14]

Erlotinib

Non-small cell lung cancer

Roche Products Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Inflammatory Conditions (n = 28)

Adalimumab

Ankylosing spondylitis

AbbVie Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Etanercept

Ankylosing spondylitis

Pfizer Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Golimumab

Ankylosing spondylitis

Janssen-Cilag Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Infliximab

Ankylosing spondylitis

Janssen-Cilag Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Adalimumab

Crohn’s disease

AbbVie Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Infliximab

Complex refractory fistulising Crohn’s disease

Janssen-Cilag Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Adalimumab

Juvenile arthritis

AbbVie Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Etanercept

Juvenile arthritis

Pfizer Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Adalimumab

Psoriatic arthritis

AbbVie Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Golimumab

Psoriatic arthritis

Janssen-Cilag Pty Ltd(

PBS

Outcome-based

Medicare Australia [14]

Infliximab

Psoriatic arthritis

Janssen-Cilag Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Pimecrolimus

Atopic dermatitis who are over 18 years of age

Novartis Pharmaceuticals Australia

PBS

Financially-based

Public Summary Document [18]

Infliximab

Crohn’s disease

Janssen-Cilag Pty Ltd

PBS

Financially-based; Outcome-based

PBPA Relativity Sheets [17], Medicare Australia [14]

Adalimumab

Complex refractory fistulising Crohn’s disease

AbbVie Pty Ltd

PBS

Financially-based; Outcome-based

PBPA Relativity Sheets [17], Medicare Australia [14]

Tocilizumab

Juvenile arthritis

Roche Products Pty Ltd

PBS

Financially-based; Outcome-based

PBPA Relativity Sheets [17], Medicare Australia [14]

Abatacept

RA

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Infliximab

RA

Janssen-Cilag Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Lu et al. 2007 [33], Pugatch et al. 2010 [21], Medicare Australia [14]

Rituximab

RA

Roche Products Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Lu et al. 2007 [33], Pugatch et al. 2010 [21], Medicare Australia [14]

Tocilizumab

RA

Roche Products Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Certolizumab Pegol

RA

UCB Australia Proprietary Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Adalimumab

RA

AbbVie Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Lu et al. 2007 [33], Pugatch et al. 2010 [21], Medicare Australia [14]

Golimumab

RA

Janssen-Cilag Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Etanercept

RA

Pfizer Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Lu et al. 2007 [33], Pugatch et al. 2010 [21], Medicare Australia [14]

Adalimumab

Severe chronic plaque psoriasis

AbbVie Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Lu et al. 2007 [33], Pugatch et al. 2010 [21], Medicare Australia [14]

Etanercept

Severe chronic plaque psoriasis

Pfizer Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Infliximab

Severe chronic plaque psoriasis

Janssen-Cilag Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Ustekinumab

Severe chronic plaque psoriasis

Janssen-Cilag Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Etanercept

Severe chronic plaque psoriasis under 18

Pfizer Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Infectious Diseases (n = 7)

Abacavir

HIV infection

ViiV Healthcare Pty Ltd

PBS

Financially-based

Robertson et al. 2009 [19], Adamski et al. 2010 [20]

Tipranavir

HIV infection

Boehringer Ingelheim Pty Ltd

PBS

Financially-based

PBS Schedule [16], Public Summary Document [18]

Entecavir

Chronic hepatitis B in adults 16 years and older with evidence of active liver inflammation

Bristol-Myers Squibb Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Boceprevir

Chronic genotype 1 hepatitis C infection

Merck Sharp & Dohme (Australia) Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Telaprevir

Chronic genotype 1 hepatitis C

Janssen-Cilag Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Posaconazole

Invasive fungal infections, not responsive to or intolerant of, alternative therapy

Schering-Plough Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Posaconazole

Prophylaxis of invasive fungal infections among high risk patients

Schering-Plough Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Pulmonary Hypertension (n = 7)

Sildenafil

PPH or PAH

Pfizer Australia Pty Ltd

PBS

Outcome-based

PBS Schedule [16], Medicare Australia [14]

Tadalafil

PPH or PAH

Eli Lilly Australia Pty Ltd

PBS

Outcome-based

PBS Schedule [16], Medicare Australia [14]

Treprostinil Sodium

PPH or PAH

Orphan Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Ambrisentan

PPH or PAH

GlaxoSmithKline Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Bosentan

PPH or PAH

Actelion Pharmaceuticals Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], PBPA Relativity Sheets [17], Public Summary Document [18], Medicare Australia [14]

Epoprostenol

PPH or PAH

GlaxoSmithKline Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], PBPA Relativity Sheets [17], Public Summary Document [18], Medicare Australia [14]

Iloprost trometamol

PPH or PAH

Bayer Australia Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Other (n = 27)

Modafinil

Narcolepsy

bioCSL (Australia) Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Verteporfin

Age-related macular degeneration

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Outcome-based

Medicare Australia [14]

Deep brain stimulation

Patients with Parkinson's disease no longer responsive to drug therapy

Not specified

MBS

Evidence generation

Stafinski et al. 2010 [15]

EVAR

Abdominal aortic aneurysm

Not specified

MBS

Evidence generation

Stafinski et al. 2010 [15]

Dabigatran

Prevention of stroke or systemic embolism in patients with non-valvular atrial fibrillation

Boehringer Ingelheim Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Atomoxetine Hydrochloride

ADHD diagnosed between the ages of 6 and 18 years

Eli Lilly Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Fingolimod

RRMS

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Financially-based; Outcome-based

Public Summary Document [18]

Cinacalcet Hydrochloride

End stage renal disease receiving dialysis who have uncontrolled secondary hyperparathyroidism

Amgen Australia Pty Ltd

PBS

Financially-based

PBS Schedule [16], Public Summary Document [18]

Clostridium botulinum type A toxin-haemagglutinin complex

Severe spasticity of the upper limb in adults following a stroke, as an adjunct to physical therapy

Ipsen Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Fentanyl citrate

Fentanyl lozenges for the treatment of breakthrough pain

Orphan Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Natalizumab

RRMS

Biogen Idec Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Paliperidone

Schizophrenia

Janssen-Cilag Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Botulinum toxin type a purified neurotoxin complex

Moderate to severe spasticity of the upper limb in adults following a stroke as an adjunct to physical therapy

Allergan Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Tenofovir Disoproxil Fumarate

Treatment of chronic hepatitis B

Gilead Sciences Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Pramipexole hydrochloride

Idiopathic Parkinson disease

Boehringer Ingelheim Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Tobramycin

Pseudomonas aeruginosa respiratory infection in a patient with Cystic Fibrosis

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Ticagrelor

ACS, MI, or unstable angina

AstraZeneca Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Pregabalin

Neuropathic pain

Pfizer Australia Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Rivaroxaban

Acute symptomatic DVT

Bayer Australia Ltd

PBS

Financially-based

Public Summary Document [18]

Aztreonam

Pseudomonas aeruginosa infection in patients with cystic fibrosis

Gilead Sciences Pty Ltd

PBS

Financially-based

Public Summary Document [18]

Crinone (progesterone gel)

Supplement progesterone in women who have luteal phase defect

Merck Serono

PBS

Financially-based

Robertson et al. 2009 [19], Adamski et al. 2010 [20]

Deferasirox (Exjade)

Chronic iron overload in patients with disorders of erythropoiesis

Novartis

PBS

Financially-based

Robertson et al. 2009 [19], Adamski et al. 2010 [20]

Omalizumab

Uncontrolled severe allergic asthma

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Aflibercept

Age-related macular degeneration

Bayer Australia Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

Ranibizumab

Age-related macular degeneration

Novartis Pharmaceuticals Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], PBPA Relativity Sheets [17], Public Summary Document [18], Medicare Australia [14]

Eltrombopag

Severe chronic immune idiopathic thrombocytopenic purpura

GlaxoSmithKline Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBPA Relativity Sheets [17], Medicare Australia [14]

Romiplostim

Severe chronic immune idiopathic thrombocytopenic purpura

Amgen Australia Pty Ltd

PBS

Financially-based; Outcome-based

PBS Schedule [16], Medicare Australia [14]

ACS = Acute Coronary Syndromes; ALL = Acute Lymphoblastic Leukemia; AML = Acute Myeloid Leukemia; CML = Chronic Myeloid Leukemia; DVT = Deep Vein Thrombosis; EVAR = endovascular aneurysm repair; GIST = Gastrointestinal Stromal Tumor; MBS = Medicare Benefits Schedule; mCRC = Metastatic Colorectal Cancer; MI = Myocardial Infarction; PAH = Pulmonary Arterial Hypertension; PBS = Pharmaceutical Benefits Scheme; PPH = Primary Pulmonary Hypertension; RA = Rheumatoid Arthritis; RCC = Renal Cell Carcinoma; RRMS = Relapsing-Remitting Multiple Sclerosis; SCCHN = Squamous Cell Carcinoma of the Head and Neck.

Apart from Australia, we found 5 schemes in New Zealand and 3 in South Korea. Table 3 summarizes the schemes [22-25]. These countries also have established a formal process for health technology assessment. Similar to Australia, New Zealand has a national health insurance for its people. The pharmaceutical management agency was established in 1993 to make decisions about drug coverage. The agency considers a number of factors, including the clinical need, clinical benefits and risks, cost-effectiveness, and budget impact of new drugs [26]. South Korea is the first country in Asia to officially adopt economic evaluation for making drug reimbursement decisions since January 2008. The Health Insurance Review Agency is responsible for reviewing the cost-effectiveness and budget impacts of new drugs for reimbursement under South Korea’s National Health Insurance [27].
Table 3

Identified patient access schemes in the Asian-Pacific region (excluding Australian examples)

Country

Drug

Indication

Company

Payer

Type

Details

Source

South Korea

Migraine medications

Migraine headaches

Not specified

National health insurance

Financially-based

Pharmaceutical companies maintained their PLS status via voluntary price cuts

Lee et al. 2012 [22]

Hypertension medications

Hypertension

Not specified

National health insurance

Financially-based

285 of the 1226 hypertension drugs instituted price reductions following reassessment of clinical usefulness & price. Drugs were delisted if they failed to show a level of clinical usefulness or if their prices were higher than 80 percentile of the highest price among drugs containing the same ingredients. If the company accepted price cuts, the price was to be lowered to the level of the 80 percentile within 3 years

Lee et al. 2012 [22]

Hyperlipidemia medications - therapeutic class

Hyperlipidemia

Not specified

National health insurance

Financially-based

Pharmaceutical companies maintained their PLS status via voluntary price cuts

Lee et al. 2012 [22]

New Zealand

Beta-interferon products, glatiramer acetate

Multiple sclerosis

Bayer

PHARMAC

Financially-based

Extended coverage for a specified number of patients

Raftery 2008 [23]

Trastuzumab

Breast cancer

Roche

PHARMAC

Financially-based

Established separate hospital program to fund distribution of cancer drugs

Raftery 2008 [23]

Imatinib

Chronic myeloid leukemia

Novartis

PHARMAC

Financially-based

Offered overall price reduction

Raftery 2008 [23]

Atorvastatin

Hypertension

Pfizer

PHARMAC

Financially-based

Price volume agreements; manufacturer committed to pay for the drug if the sales exceed a fixed threshold

Antonanzas et al. 2011 [24]

 

Adalimumab

Arthritis

Abbott Laboratories NZ Ltd

PHARMAC

Financially-based

Adalimumab spending is probably overstated due to a risk sharing agreement between the sponsor and PHARMAC, which involves rebates paid by the sponsor once Government spending reaches a certain level

Access Economics report for Arthritis New Zealand 2010 [25]

HIRA = Health Insurance Review & Assessment Service; PLS = positive list system; PHARMAC = The Pharmaceutical Management Agency.

Types of medical products

Almost all patient access schemes focused on pharmaceuticals (97.2%; n = 103), few on medical technologies. More than half of schemes covered treatments for non-communicable diseases, predominantly cancer (n = 31) and inflammatory diseases (n = 29) such as rheumatoid arthritis. Schemes in Australia were largely established for technologies with high budget impact due to high cost per patient (e.g., adalimumab, which costs approximately $20,000 per patient-year) or large volumes of use (e.g., dabigatran for prevention of stroke or systemic embolism), as well as for products that may be used beyond their approved indications (e.g., entecavir that is subsidized only for chronic hepatitis B in adults with evidence of active liver inflammation). Similarly, New Zealand targeted new products with high cost per-patient or large volumes of use. Instead of individual drugs, the schemes in South Korea targeted the entire class of older medications with high budget impact due to large volumes of use (e.g., hyperlipidemia medications).

Outcome-based schemes

Nearly two-thirds of schemes in Australia (63.3% n = 62; Table 2) included a “conditional treatment continuation” component that limits continued subsidy of high-cost medicines to patients who demonstrate an adequate clinical response. These schemes specify strict criteria for both initial and continued access. For example, the scheme for adalimumab for rheumatoid arthritis requires confirmation of clinical improvement at 3 months (measured by reductions in the total number of affected joints and in levels of inflammation markers) for continued subsidy [28]. In the scheme for bosenten in Australia, price was directly linked to the observed survival of patients treated with bosentan to confirm the clinical benefits (i.e., survival) and cost-effectiveness assumed at the time of assessment [29].

Evidence generation schemes

We found three schemes with an evidence generation component in Australia, all focused on medical technologies: positron emission tomography, deep brain stimulators, and endovascular aneurysm repair (Table 2). In all three cases, the Australian government agreed to provide interim funding for access to the technology and to support collection of relevant clinical (and cost) information [15]. The manufacturers did not share the financial costs to generate evidence.

Financially-based schemes

Financially-based schemes involving price reductions, price-volume agreements, or utilization caps were the most common risk-sharing schemes across the Asia-Pacific region (77.4%; n = 82). We identified 3 in South Korea, 5 in New Zealand, and 74 schemes in Australia. Forty-one of 74 schemes in Australia were hybrid schemes that involved both pricing arrangements and conditional treatment continuation.

Discussion

In attempts to provide access to promising technologies, many countries are assessing the potential applicability of patient access schemes for their markets; these schemes typically involve novel arrangements between payers and manufacturers. We reviewed the literature on patient access schemes in the last two decades, with a focus on experiences in the Asia-Pacific region. We found a few schemes from South Korean and New Zealand, and many more schemes in Australia. Though different in their development and implementation, these schemes have arisen in response to cost pressures, demands from key stakeholders, and the inherent uncertainties in the health value and estimated budget impact of medical products in real-world settings. Pharmaceuticals account for nearly all identified schemes. The preponderance of pharmaceutical schemes may reflect the different evidence profiles at product launch, the higher budget impact of pharmaceuticals versus devices, and/or the different intellectual property and patent environments. There do not appear to be guidelines on when patient access schemes are applied or which type of scheme is most appropriate in a given circumstance. However, our findings suggest that the main targets are pharmaceuticals likely to have high budget impact due to high per-patient acquisition costs and/or potential large volumes of use, and pharmaceuticals likely to be used beyond their approved indications.

In the Asia-Pacific region, Australia has the most experience with patient access schemes. This may reflect the negotiating power and extensive experience in technology assessment, due to the design of the regulatory, institutional, and policy structures for medicines benefit decisions in the Australian publicly-funded national healthcare system [4,6]. High-cost medicines for treatment of cancer and inflammatory diseases were the targets of more than half of the schemes in Australia. Typical outcome-based schemes, in which the price or level of reimbursement is tied to achieving intermediate or final clinical endpoints, have rarely been used in Australia, with bosentan being the only example. Implementing outcome-based schemes is complex: they require detailed longitudinal information on patient clinical status (e.g., disease severity and progression, comorbidities) [30]; they require substantial financial, human, and infrastructure resources for monitoring of patients and financial transactions related to treatments [31]; and they require a mechanism for adjusting price or level of reimbursement when explicit clinical endpoints are not reached. Instead, Australia limits continued subsidy of high-cost medicines to patients who demonstrate an adequate response, known as “conditional treatment continuation” policies, which are often coupled with pricing arrangements, that is, hybrid schemes. Initial access is also restricted to a small pool of patients largely based on disease severity and non-responsiveness to less expensive therapeutic alternatives. Criteria restricting both initial and continued access aim to maximize value-for-money and control costs. While some evidence suggests that such policies can cap spending [28,32], whether they achieve value-for-money or if patients who need the medicines actually gained access should be investigated. Further, strict initial and continued access criteria are ethically challenging as individual patients may just missed the arbitrary threshold for access [33,34].

Evidence generation schemes were also rarely used in the Asia-Pacific region, possibly due to the complexity in tracking patient outcomes and the additional costs and administrative burden involved to operate these schemes.

In contrast, we found that financially-based schemes were common, possibly due to fewer operational challenges. Price-volume agreements and utilization caps have been in place for more than a decade in Australia [19]. Typically, product prices are reduced if sales exceed pre-agreed volumes, or expenditures refunded by the manufacturer if government expenditures exceed a pre-agreed cap or threshold. Price-volume agreements can shift cost considerations from the payer to the manufacturer, which is important especially if there are concerns that (i) new medicines will be prescribed to a wider population than envisaged, (ii) the patients prescribed the drug will not always be those most likely to gain the greatest benefit [4,11,35], and/or (iii) the product does not result in the expected clinical benefits. Expectations are that manufacturers will target promotion in accordance with approved prescribing requirements. Details of patient access schemes (e.g., capped prices, negotiated volumes) are generally unavailable to the public [19].

Our study has several limitations. The review focused on the Asia-Pacific region; however, the investigators were limited to the English language literature; schemes that were described only in other languages have not been included. Given the sensitive nature of contracts in this field, it is likely information on schemes remains unpublished. Manufacturers will be reluctant to disclose details about patient access schemes if such information will be available to other countries that use external reference pricing. We thus are likely to have underestimated the total number of patient access schemes in Asia-Pacific countries. Nevertheless, this study provides insights about what types of products are commonly targets for patient access schemes and about recent experiences in the Asia-Pacific region that may inform the development of future schemes.

Although beyond the scope of this review, we noted that industry-sponsored patient assistance programs were implemented in several countries to improve access to medicines. For instance, the Glivec International Patient Assistance Program, implemented since 2001 in 81 low and middle-income countries (including China, India, Indonesia, Thailand, and Vietnam), has provided access to imatinib for patients with specific types of leukemia or gastrointestinal cancer [36]. The MUSANDA patient assistance program, implemented in the United Arab Emirates, supports access to ranibizumab for age-related macular degeneration. Although payers are usually not involved in industry-sponsored patient assistance programs, these programs can provide interim access to innovative medicines while payer-industry patient access schemes are under negotiation; they can complement payer-industry patient access schemes by providing treatments for individual patients who are unable to self-fund treatments and who have no third-party payer; or they can become a vehicle for implementing a payer-industry patient access scheme, as has happened for coverage of imatinib in Thailand [37].

Globally, forecasts estimate that spending on new biologic agents will outpace overall spending growth on medicines and represent about 20% of the estimated US$1.2 trillion total pharmaceutical market by 2017 [38]. The recent availability of sofosbuvir, a new, curative treatment for hepatitis C, a disease that is highly prevalent in many low and middle-income countries, highlights the need for innovation in medicines financing. Refined patient access schemes may constitute much-needed innovations in financing arrangements that provide equitable and affordable access for those who need the new medicines. Payers have an incentive to develop strategies that can help control costs while ensuring patient access to medical products that benefit health. Patient access schemes may also mitigate the negative impacts of uncertainties in cost-effectiveness and budget impact estimates, and shift payer and manufacturer focus to improving patient outcomes in real-world settings – the ultimate goal of medical care. Payers have different ranges of authority over pricing, access, and evidence generation; some are more limited in the types of coverage decisions or access schemes they can consider, which may explain some of the variation in use of schemes between countries. Manufacturers are likely to prefer patient access schemes over a denial of coverage or explicitly reduced pricing, in part because patient access schemes can keep the real reimbursement prices confidential allowing them to tier prices by markets without the threat of external reference pricing [5,13].

There is little evidence on whether patient access schemes achieve their intended goals; they are a relatively recent development and the details of such agreements are usually confidential. However, key stakeholders appear willing to at least discuss types of schemes that can enable patient access to needed medicines [4,31,33]. Future studies should generate evidence on whether patients who need the medicines actually receive and benefit from them; whether schemes make high quality care more affordable for households and systems; whether they provide incentives for manufacturers to continue to invest in products that meet unmet needs; and whether data collected as part of the schemes confirm estimated cost-effectiveness or long-term benefits. Such evidence about potential benefits and costs is needed to inform the adoption of patient access schemes globally. Other factors that may affect their adoption center around operational challenges such as administrative burden and difficulty in tracking patient outcomes in healthcare delivery and financing systems [4,5,13,28,30-34,39], as well as the transparency and perceived fairness of the complex decisions on the establishment of patient access schemes and criteria for access [33,34]. Research is needed to compare costs of administration and operation on the payer side and effects on pricing strategies globally on the pharmaceutical company side between patient access schemes and upfront discounts in drug prices.

Conclusions

Patient access schemes offer an important option for healthcare systems to allow patient access to promising technologies that may not otherwise be funded. Our study adds to existing knowledge by identifying and characterizing published patient access schemes in the Asia-Pacific region. Financially-based access schemes are most common. Australia tends to couple conditional treatment continuation with financial arrangements to provide further assurance. The main targets of patient access schemes are pharmaceuticals likely to have high budget impact due to high per-patient costs and/or large volumes of use, and pharmaceuticals that may be adopted more widely than indicated. With the rapid proliferation of high-cost medicines, these schemes may increasingly be used to enable access to innovative care within finite budgets. Future research is needed to generate evidence about the effectiveness and economic, administrative, and company pricing policy consequences of patient access schemes.

Authors’ Affiliations

(1)
Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute
(2)
Jefferson Medical School
(3)
School of Pharmacy, University of Auckland

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Copyright

© Lu et al.; licensee BioMed Central. 2015

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